Research visit: Tim Tree

My Name’5 Doddie Foundation is supporting Professor Timothy Tree at King’s College London on an exciting project to explore a new potential treatment for MND that works by harnessing the body’s own immune system. Our Director of Research, Jessica Lee, and Director of Fundraising, Paul Thompson, visited Tim to learn more about his research and the impact it could have for people with MND.

The immune system and MND

Our immune systems are made up of a complex mix of different cell types that work together to fight infections. Sometimes, some of these cells malfunction and cause the immune system to produce an immune response, even in the absence of an infection. This can cause accidental damage within the body, such as inflammation in the joints associated with rheumatoid arthritis. To prevent this malfunction, a specific type of immune cell called a “regulatory T cell” keeps other immune cells in check and ensures the immune system is functioning correctly.People with MND have problems with their regulatory T cells; they are either reduced in number or they don’t work as well as they should and this leads to an increased immune response, compared to people without MND. This could contribute to the progression (but not the cause) of MND, by the overactive immune system accidentally damaging motor neurons. Treatments that increase the number and function of regulatory T cells could therefore be a promising prospect for the treatment of MND.

A potential MND treatment

IL-2 (also known as interleukin-2) is a naturally occurring molecule in the body that has been shown to increase the number of regulatory T cells. IL-2 has already been tested in a number of diseases including type 1 diabetes and other autoimmune diseases; we therefore know it is safe and tolerable in humans.A phase 2 trial in people with MND, called “IMODALS”, showed that low dose IL-2 successfully increased the number of regulatory T cells in a small number of people with MND. Because of this promising result, a larger trial, called “MIROCALS” was carried out to assess whether low doses of IL-2 improved outcomes for people with MND. The results of MIROCALS are not yet published, so we do not currently know whether it is effective at delaying survival or slowing progression of the disease.

How Tim’s research fits in

Participants in the MIROCALS trial generously consented to provide 4 blood samples and 3 cerebral spinal fluid (CSF) samples over the course of the study. These samples provide researchers with a huge amount of information about how each individual responded to the treatment. It could help them to understand why some people might respond better than others, so that we can predict which people would benefit most from the treatment in the future.As part of this project funded by My Name’5 Doddie Foundation, Tim and his team are working to analyse the precious samples from the MIROCALS trial. The majority of the work is carried out by a postdoctoral scientist in Tim’s lab, Dr Yoana Arroyo. The team will assess how treatment with low dose IL-2 affected the number and behaviour of regulatory T cells in people with MND. This information will then be linked to how each patient responded to the treatment to provide a better understanding of the role of regulatory T cells in MND.

The impact this research could have

This project will provide valuable insights on how the power of the immune system can be harnessed to tackle MND, and whether IL-2 could be a good treatment option. Tim, Yoana and the rest of the team also hope that the work could shed light on other potential treatment strategies, working towards our goal of finding treatments that will slow, stop or reverse the progression of MNDMy Name’5 Doddie Foundation is grateful to Tim and his team for their commitment to this project, and to our supporters for their generosity that has allowed us to fund this important work. Mostly, we would like to thank the people with MND who took part in the MIROCALS trial.